On June 4, 2025 Acerand Therapeutics, a clinical-stage biotech company focusing on the discovery and development of innovative small-molecule therapies in oncology, reported the dosing of the first patient in its first-in-human Phase I clinical trial (NCT06801236) of ACE-232, a novel oral inhibitor of CYP11A1 (Press release, Acerand Therapeutics, JUN 4, 2025, View Source [SID1234653728]). The trial is being conducted in patients with metastatic castration-resistant prostate cancer (mCRPC).

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This multicenter Phase I study is being conducted in both the United States and China. The study comprises two parts: a dose-escalation phase (Phase IA) and a dose-optimization phase (Phase IB). The primary objectives are to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), as well as preliminary clinical activity of ACE-232, and to determine the recommended Phase II dose (RP2D). Professor Emmanuel Antonarakis, Director of Genitourinary Oncology at Masonic Cancer Center, University of Minnesota, is serving as the global coordinating Principal Investigator for the study. "I would like to congratulate the Acerand team, the research staff, and the first patient for helping us to reach this important milestone," says Dr. Antonarakis.

Preclinical data demonstrated that ACE-232 possesses superior potency, efficacy, and pharmacokinetic properties (flat PK curve and long half-life profile) compared to other investigational CYP11A1 inhibitors, including MK-5684 (Opevesostat, formerly known as ODM-208). ACE-232 showed excellent tolerability in preclinical models, presenting a wide therapeutic window for clinical development.

The initiation of this trial marks a significant milestone for Acerand, representing its first clinical study in the United States and highlighting its strategic commitment to the global development of innovative cancer therapies that address major unmet medical needs.

About ACE-232

ACE-232 is a highly potent and selective small-molecule inhibitor of CYP11A1, a key adrenal enzyme involved in the first and rate-limiting step of steroid hormone biosynthesis. By targeting CYP11A1, ACE-232 aims to suppress the production of androgens and other steroid hormones, offering a novel therapeutic mechanism for the treatment of androgen-dependent (including enzalutamide or abiraterone-resistant) prostate cancer.

On June 4, 2025 Rakuten Medical, Inc., a global biotechnology company developing and commercializing Alluminox platform-based photoimmunotherapy, reported that its third drug candidate, RM-0256, has been selected for funding by the Japan Agency for Medical Research and Development (AMED) under its Support Program for Orphan Drug prior to the Designation (Press release, Rakuten Medical, JUN 4, 2025, View Source [SID1234653727]).

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RM-0256 is a novel antibody conjugate composed of IRDye700DX (IR700)—a light-activatable dye for which Rakuten Medical holds exclusive global manufacturing and supply rights—and a proprietary anti-PD-L1 monoclonal antibody. The AMED funding will support the clinical development of RM-0256 photoimmunotherapy for unresectable, advanced, or recurrent malignant epithelial tumors.

In Japan, malignant epithelial tumors affect approximately 22,000 individuals annually1. While systemic therapies, including chemotherapy and immune checkpoint inhibitors, are currently the mainstay of treatment for unresectable advanced or recurrent cases, patients who develop resistance to these treatments face limited treatment options, which underscores the urgent need for novel therapeutic approaches.

RM-0256 targets Programmed Cell Death Ligand 1 (PD-L1), a key immune checkpoint protein that allows tumors to evade immune detection by binding to PD-1 receptors on activated T cells. PD-L1 is widely expressed in various solid tumors—including melanoma, ocular melanoma, lung, urothelial, gastrointestinal, gynecological, breast, and head and neck cancers2—as well as on immunosuppressive cells within the tumor microenvironment.

Pre-clinical studies of PD-L1-targeted photoimmunotherapy have suggested three complementary mechanisms of action3:

1) Direct depletion (necrosis) of PD-L1-expressing tumor cells;
2) Activation of anti-tumor immunity through the elimination of PD-L1–expressing immunosuppressive cells;
3) Checkpoint blockade, by inhibiting PD-L1/PD-1 interaction, potentially enhancing systemic immune responses.

With the support of AMED funding, Rakuten Medical is accelerating the development of RM-0256 photoimmunotherapy as a novel, multimodal cancer therapy that may induce both local and systemic anti-tumor effects.

On June 4, 2025 Duality Bio (HKEX: 9606.HK) reported the preliminary data of two clinical trials, HER3 ADC candidate DB-1310 and B7H3 ADC candidate DB-1311/BNT324, which is being jointly developed with BioNTech, in Oral/Rapid Oral presentations at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting taking place in Chicago, US, from May 30 to June 3 (Press release, DualityBio, JUN 4, 2025, View Source [SID1234653726]).

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B7H3 ADC candidate DB-1311/BNT324:

Data from the ongoing Phase 1/2 clinical trial (NCT05914116) in patients with heavily pre-treated castration-resistant prostate cancer (CRPC) were presented during an oral session on BNT324/DB-1311, an B7H3-targeted ADC candidate.

The data indicated early clinical activity and a manageable safety profile with low discontinuation rates. Most common adverse events were gastrointestinal and hematologic toxicities. In the 52 efficacy evaluable patients with heavily pretreated CRPC based on RCIST v1.1, the confirmed objective response rate (cORR) was 30.8%, DCR was 90.4%. Among 68 evaluable patients, the 6-month rPFS rate was 69.8%. Similar outcomes were observed across both dose levels (6 mg/kg or 9 mg/kg). Outcomes appeared better in earlier treatment lines and in patients who received one prior NHT, while antitumor activity was also observed in later lines and regardless of type of prior treatment or metastatic site.

The clinical trial is currently enrolling post Lu-177 CRPC (Cohort 11) and taxane-naïve CRPC (Cohort 12).

As the incidence rate of prostate cancer is increasing[1],1 there is a high unmet need for new effective therapies for patients with heavily pretreated CRPC[1]. The DB-1311/BNT324 program received Fast Track Designation by the U.S. Food & Drug Administration ("FDA") for the treatment of patients with advanced/unresectable or metastatic CRPC that has progressed on or after standard systemic regimens in 2024.

HER3 ADC DB-1310：

Data from the first-in-human Phase I/IIa study (NCT05785741) presented by Professor Aaron E. Lisberg of the University of California, Los Angeles (UCLA), demonstrated that DB-1310 showed encouraging efficacy and a manageable safety profile in patients with advanced solid tumors who had failed standard treatments.

Among 123 efficacy evaluable patients, the unconfirmed objective response rate (uORR) was 31%, and the disease control rate (DCR) reached 84%. Notably, efficacy was particularly striking in the key subgroup of patients with EGFR-mutated non-small cell lung cancer (NSCLC) (n=46) where uORR reached 44%, DCR was 91%, median progression-free survival (mPFS) was 7.0 months, and median overall survival (mOS) was 18.9 months. The uORR reached an impressive 66.7% at the 5.5 mg/kg Q3W dose level (n=12).

In addition, DB-1310 was well-tolerated with a manageable safety profile. The most common treatment-related adverse events (TRAEs) were Grade 1-2 hematological and gastrointestinal events with a low treatment-related discontinuation rate of 3.5%.

These positive results support the continued development of DB-1310 in advanced solid tumors, particularly in patients with EGFR-mutated NSCLC. The company is advancing its global development program, including exploring DB-1310 as a monotherapy in additional tumor types, as well as exploring DB-1310 in combination with EGFR TKIs and HER2-targeted therapies.

On June 4, 2025 Veeva Systems (NYSE: VEEV) and Sarah Cannon Research Institute (SCRI) reported a strategic collaboration to drive speed and efficiency in oncology clinical trials across SCRI’s more than 200 research site locations (Press release, Sarah Cannon Research Institute, JUN 4, 2025, View Source [SID1234653725]). SCRI is adopting Veeva Clinical Platform to unify its contract research organization (CRO) and site management organization (SMO) on a single platform for seamless data flow across clinical teams and research sites.

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"We are thrilled to advance our clinical trials by integrating Veeva Clinical Platform into our digital toolkit," said SCRI chief information and digital officer Yazhene Krishnaraj. "This strategic collaboration empowers our clinical teams to deliver groundbreaking therapies to patients with enhanced precision and speed."

Veeva Clinical Platform will enable SCRI to simplify and standardize trial processes and information flow, improving how investigators and clinical teams work together and share data. With a connected foundation for clinical research, SCRI will be able to automate key processes and provide a streamlined experience for its sites.

"We’re excited to work closely with SCRI to drive innovation in oncology research," said Jim Reilly, president of Veeva Development Cloud. "In a first-of-a-kind partnership, Veeva Clinical Platform will serve as SCRI’s clinical trial foundation for its CRO and SMO. By standardizing operations on one platform, we can support SCRI in delivering faster and more cost-effective trials."

On June 4, 2025 Continuity Biosciences, LLC, a developer of advanced drug delivery technologies, reported the acquisition of Focal Medical, Inc., a North Carolina-based biopharmaceutical company pioneering site-specific chemotherapy using iontophoresis (Press release, Continuity Biosciences, JUN 4, 2025, View Source [SID1234653724]). The acquisition represents a key step in Continuity’s strategy to become a leader in device targeted therapeutics for intractable solid tumors.

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Founded by leading scientists and clinicians, Focal Medical has developed a proprietary platform that delivers chemotherapy directly to tumors using iontophoresis—this minimally-invasive technique utilizes a mild electrical current to drive therapeutic compounds into targeted tissues. This method enhances local drug concentration while minimizing systemic toxicity, a critical need in the treatment of pancreatic and other solid-tissue cancers.

Focal Medical’s lead product candidate, an iontophoresis device directed gemcitabine therapy for pancreatic cancer, has been cleared by the U.S. Food and Drug Administration (FDA) through an Investigational New Drug (IND) application. Phase 1b clinical trials are expected to begin later this year.

"This is a transformative milestone for Continuity Biosciences," said Ramakrishna Venugopalan, PhD, MBA, Chief Executive Officer of Continuity Biosciences. "Focal Medical’s approach to localized drug delivery aligns perfectly with our vision to improve bioavailability, reduce systemic exposure, and deliver better outcomes for patients. We are thrilled to bring this promising platform into our portfolio and into the clinic."

Through the acquisition, Continuity Biosciences obtains Focal Medical’s entire patent estate, iontophoresis technology platform and specialized equipment, scientific know-how, and licensing agreements with the University of North Carolina at Chapel Hill. In addition, key scientific staff and leadership from Focal will join Continuity, further enhancing its internal capabilities. A dedicated research and development facility in Cary, NC that supports ongoing platform and pipeline advancement is also part of the acquisition and will be expanded to support other delivery platforms.